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Cambridge lab producing drug that could make history in fight against ALS

A lab in Cambridge is producing a drug that could make history in the fight against amyotrophic lateral sclerosis, or ALS, in part from money that was raised during the Ice Bucket Challenge in 2014.

“This has never happened before,” says Carol Hamilton, senior director of development at the ALS Therapy Development Institute. “Where a non-profit has discovered and developed a drug that they’ve brought forward into the clinic for patients to try.”

The ALS Therapy Development Institute (ALS TDI) has been testing that drug, known as AT-1501, on mice. The drug slows down the progression of the devastating neurological disease.

CEO Steve Perrin says the results are promising.

“If you get diagnosed with a horrible disease that’s progressive and an effective treatment slows it down, you’ll be able to see your kids go to college,” Perrin says. “You’ll be able to see your kids grow up, get married, have their own kids. That’s really what the goal of drug development initially is.”

The lab is now waiting to hear from the FDA to approve human trials, hoping to get it into people by the end of the year.

It took $8.5 million to get the development of the drug to this point. A good chunk of that money came from the Ice Bucket Challenge, the viral sensation co-founded by Beverly’s Pete Frates, a former Boston College baseball player diagnosed with ALS in 2012. The Frates family once again took part in that event over the weekend in their hometown.

There are also fundraisers like ALS TDI’s “Tri-State Trek,” a 280-mile bike ride from Boston to Connecticut in June.

“This is a big piece of what we do every year,” says Hamilton. “This is not only going to help AT-1501, but we have other drugs coming up behind it that we need to move forward.”

7News’ Jadiann Thompson was among those who hit the road, riding the Trek in honor of her grandmother, JoAnn, who lost her battle with ALS in 1994.

Hundreds of people ride for a reason including the lab’s scientists and CEO.

“Because of who we are and because of the commitment of our community, we all step up to try to help as much as we can supporting our cause,” says Perrin.

He says it’s the people battling the disease that inspire everyone to work as hard as they can.

“It makes you get up at 4 a.m.,” says Perrin. “It makes you work all day long and we’re not going to stop until we find a cure.”